Myelodysplastic Syndrome
Conditions
Keywords
MDS
Brief summary
Multi-center, survival data collection in subjects previously enrolled in study NCT00065156 (Celgene Protocol CC-5013-MDS-003).
Interventions
None listed
Sponsors
Celgene
Study design
Observational model
OTHER
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Must have been enrolled in the Celgene protocol CC-5013-MDS-003 study. 2. Must understand and be able to give informed consent (if a subject is deceased, proper legal consent (i.e. next of kin, legal representative) will be obtained prior to collection of data).
Exclusion criteria
1\. Consent refused for any reason at current or long-term follow up completed in 2007 (long-term follow up: survival data collection completed to obtain further safety information on CC-5013-MDS-003 discontinued subjects from May - October 2007).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Participants Survival Status as of the Time of the Extension Study Follow-up | up to 7 years | Count of participants who were alive or deceased at the time of the extension study follow-up. |
| Kaplan Meier Estimate for Overall Survival | up to 7 years | Overall survival was measured from the start of therapy in CC-5013-MDS-003 to the date of death from any cause. Results include data collected during the extension follow-up. |
| Participants Status Regarding Progression to Acute Myeloid Leukemia (AML) as of the Time of the Extension Study Follow-up | up to 7 years | Count of participants who progressed to AML at the time of the extension study follow-up. |
| Kaplan Meier Estimate for Progression to Acute Myeloid Leukemia (AML) | up to 7 years | Progression to AML was measured from the start of therapy in CC-5013-MDS-003 to the date AML was diagnosed. Results include data collected during the extension follow-up. |
| Cause of Death for Participants Who Died | up to 7 years | Summary of the cause of death for participants from MDS-003 who died as of the time of the extension study follow-up. |
Countries
Germany, United States
Participant flow
Recruitment details
This extension study was conducted specifically to provide further long-term outcomes as regards overall survival/vital status and the possible occurrence of progression to AML for all participants previously enrolled in study NCT00065156 (Celgene CC-5013-MDS-003). Participants from the original study are included in the Participant Flow.
Pre-assignment details
When the final MDS-003 clinical study report was written, 76 participants had died; therefore, 72 of all 148 participants who first enrolled in the MDS-003 study could have been included in the extension study. Sixteen did not participate because their investigative sites did not participant. Two withdrew consent during the earlier study.
Participants by arm
| Arm | Count |
|---|---|
| Lenalidomide No intervention was given during this extension study which gathered survival information on participants of study NCT00065156 (Celgene study CC-5013-MDS-003). During the CC-5013-MDS-003 study, participants initially took a syncopated dosage regimen in which 10 mg of lenalidomide was taken orally once daily on Days 1 to 21 of a 28-day cycle. The study was amended to employ a continuous dosage regimen in which 10 mg of lenalidomide was taken without a planned rest period. Participants who initially began therapy on the syncopated regimen and who did not experience dose-limiting adverse events (AEs) were allowed to switch to the continuous regimen. | 148 |
| Total | 148 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| MDS-003 Study | Adverse Event | 37 |
| MDS-003 Study | Death | 11 |
| MDS-003 Study | Disease Progression | 7 |
| MDS-003 Study | Investigator Decision | 2 |
| MDS-003 Study | Lack of Efficacy | 52 |
| MDS-003 Study | Loss of Response | 1 |
| MDS-003 Study | Lost to Follow-up | 1 |
| MDS-003 Study | Non-Compliance | 2 |
| MDS-003 Study | Secondary Malign Disease | 1 |
| MDS-003 Study | Study Closed by Sponsor | 2 |
| MDS-003 Study | Withdrawal by Subject | 8 |
Baseline characteristics
| Characteristic | Lenalidomide |
|---|---|
| Age, Continuous | 70.0 years STANDARD_DEVIATION 10.5 |
| Region of Enrollment Germany | 36 participants |
| Region of Enrollment United States | 112 participants |
| Sex: Female, Male Female | 97 Participants |
| Sex: Female, Male Male | 51 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 |
Outcome results
Primary
Cause of Death for Participants Who Died
Summary of the cause of death for participants from MDS-003 who died as of the time of the extension study follow-up.
Time frame: up to 7 years
Population: Safety population of participants who died
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Lenalidomide | Cause of Death for Participants Who Died | Disease progression - AML | 24 participants |
| Lenalidomide | Cause of Death for Participants Who Died | Disease progression - MDS | 7 participants |
| Lenalidomide | Cause of Death for Participants Who Died | Infection - Sepsis | 9 participants |
| Lenalidomide | Cause of Death for Participants Who Died | Infection - Respiratory | 4 participants |
| Lenalidomide | Cause of Death for Participants Who Died | Infection - Infection (not specified) | 3 participants |
| Lenalidomide | Cause of Death for Participants Who Died | Cardiac - Cardiac heart failure | 9 participants |
| Lenalidomide | Cause of Death for Participants Who Died | Cardiac - Myocardial infarction | 3 participants |
| Lenalidomide | Cause of Death for Participants Who Died | Cardiac - Sudden death | 1 participants |
| Lenalidomide | Cause of Death for Participants Who Died | Hemorrhage - Cerebral hemorrhage | 3 participants |
| Lenalidomide | Cause of Death for Participants Who Died | Hemorrhage - Gastrointestinal hemorrhage | 1 participants |
| Lenalidomide | Cause of Death for Participants Who Died | Neoplasm - Endometrial | 1 participants |
| Lenalidomide | Cause of Death for Participants Who Died | Neoplasm - Lung Cancer | 1 participants |
| Lenalidomide | Cause of Death for Participants Who Died | Neoplasm - Ovarian | 1 participants |
| Lenalidomide | Cause of Death for Participants Who Died | Other Events - Multi-organ failure | 2 participants |
| Lenalidomide | Cause of Death for Participants Who Died | Gastrointestinal - Intestinal perforation | 1 participants |
| Lenalidomide | Cause of Death for Participants Who Died | Venous-thromboembolic - Pulmonary embolism | 1 participants |
| Lenalidomide | Cause of Death for Participants Who Died | Others - Cause of death unknown | 29 participants |
| Lenalidomide | Cause of Death for Participants Who Died | Hemorrhage - Unknown origin | 1 participants |
Primary
Kaplan Meier Estimate for Overall Survival
Overall survival was measured from the start of therapy in CC-5013-MDS-003 to the date of death from any cause. Results include data collected during the extension follow-up.
Time frame: up to 7 years
Population: Intent to treat population
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Lenalidomide | Kaplan Meier Estimate for Overall Survival | 39.47 months |
Primary
Kaplan Meier Estimate for Progression to Acute Myeloid Leukemia (AML)
Progression to AML was measured from the start of therapy in CC-5013-MDS-003 to the date AML was diagnosed. Results include data collected during the extension follow-up.
Time frame: up to 7 years
Population: Intent to treat population. One participant was diagnosed by central reviewer as having AML at entry to the MDS-003 study (baseline) so was excluded from this analysis.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Lenalidomide | Kaplan Meier Estimate for Progression to Acute Myeloid Leukemia (AML) | NA months |
Primary
Participants Status Regarding Progression to Acute Myeloid Leukemia (AML) as of the Time of the Extension Study Follow-up
Count of participants who progressed to AML at the time of the extension study follow-up.
Time frame: up to 7 years
Population: Intent to treat population. One participant was diagnosed by central reviewer as having AML at entry to the MDS-003 study (baseline) so was excluded from this analysis.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Lenalidomide | Participants Status Regarding Progression to Acute Myeloid Leukemia (AML) as of the Time of the Extension Study Follow-up | Progressed to AML | 36 participants |
| Lenalidomide | Participants Status Regarding Progression to Acute Myeloid Leukemia (AML) as of the Time of the Extension Study Follow-up | Did not progress to AML | 86 participants |
| Lenalidomide | Participants Status Regarding Progression to Acute Myeloid Leukemia (AML) as of the Time of the Extension Study Follow-up | Unknown | 25 participants |
Primary
Participants Survival Status as of the Time of the Extension Study Follow-up
Count of participants who were alive or deceased at the time of the extension study follow-up.
Time frame: up to 7 years
Population: Intent to treat population
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Lenalidomide | Participants Survival Status as of the Time of the Extension Study Follow-up | Deceased | 101 participants |
| Lenalidomide | Participants Survival Status as of the Time of the Extension Study Follow-up | Alive | 29 participants |
| Lenalidomide | Participants Survival Status as of the Time of the Extension Study Follow-up | Unknown | 18 participants |